Amarantus and Lonza Walkersville, Inc. Provide Update on cGMP Manufacturing Readiness for the Engineered Skin Substitute (ESS) Program
SAN FRANCISCO, February 22, 2016 /PRNewswire/ --
Amarantus BioScience Holdings, Inc. (OTCQB: AMBS), a biotechnology company focused on developing products for Regenerative Medicine, Neurology and Orphan Diseases, today updated the status of its cGMP manufacturing technical transfer for producing ESS at Lonza Walkersville, Inc., a premier contract manufacturer providing cell and tissue-based products for clinical development.
Amarantus announced that it has successfully completed the growth and testing components of its confirmatory engineering run as previously anticipated, and is now finalizing the documentation, sterilization validation, and auditing that is required to complete the technical transfer. Once technical transfer is finished, which is expected to be in the first quarter of 2016, Amarantus will be operationally ready to supply ESS for the planned Phase 2 clinical study for the treatment of full thickness thermal burns covering over 50% of the body with the US Army. The 10-patient trial will be conducted under a Collaborative Research & Development Agreement (CRADA) with the U.S. Army's Institute for Surgical Research (ISR) at Fort Sam Houston in Texas. Two additional civilian sites will be opened to accelerate enrollment of the study.
Completion of the confirmatory engineering run is a highly significant milestone, as this is a demonstration that the ESS investigational product can be grown from adult (rather than only from pediatric) donor tissue using a scalable and fully cGMP-compliant process.
"In October 2015, the Company decided to focus the majority of its available resources on the ESS program, due largely to the incredible need for this product in the marketplace - evidenced by the US Army's financial support and strong desire to open enrollment for the upcoming Phase 2 clinical study to treat U.S. soldiers who have been critically burned serving their country," said Gerald E. Commissiong, President & CEO of Amarantus. "Today's announcement marks a major milestone towards the opening of this study. We have demonstrated that we can grow ESS, a full thickness autologous human skin graft replacement product, in a cGMP environment. Moving forward, we will be laser-focused on making certain that this breakthrough cell therapy product candidate is well positioned from a regulatory perspective to gain marketing approval as quickly as possible."
Amarantus is planning three distinct clinical development programs for ESS, all of which have the opportunity to gain rapid market approval in various orphan dermatologic conditions:
- Adult severe burns: Initiating a 10-patient Phase 2 clinical development program with US Army study under CRADA at ISR and two additional leading civilian burn centers;
- Pediatric severe burns: Evaluating Phase 3 development program designs with leading pediatric burn center(s) in the United States;
- Giant Congenital Melanocytic Nevi (GCMN): Evaluating pivotal Phase 3 development program designs with leading dermatology center(s) in the United States.
Amarantus President & CEO Gerald E. Commissiong will be giving a corporate update at 3:30pm at the SeeThru Equity & Brewer Group 2nd Annual Innovations and Investor Conference on February 22, 2016. Mr. Commissiong will discuss the company's near-term prospects, including the pending spinout of wholly-owned subsidiary Amarantus Diagnostics into Avant Diagnostics and the pending initiation of the Phase 2 clinical study with the US Army for the treatment of soldiers burned over 50% of their bodies with ESS. Mr. Commissiong's presentation will be webcast live at http://wsw.com/webcast/seethru15/ambs , and will be available online for 90 days following the presentation on the same website.
About Engineered Skin Substitute (ESS)
Engineered Skin Substitute (ESS) is a tissue-engineered skin prepared from autologous (patient's own) skin cells. It is a combination of cultured epithelium with a collagen-fibroblast implant that produces a skin substitute that contains both epidermal and dermal components. This model has been shown in preclinical studies to generate a functional skin barrier. Most importantly, because ESS is composed of a patient's own cells, it is less likely to be rejected by the immune system of the patient, unlike with porcine or cadaver grafts in which immune system rejection is a possibility. A non-GMP version ESS has been used in investigator initiated and compassionate use clinical settings in over 150 human subjects, primarily pediatric patients, for the treatment of severe burns up to 95% total body surface area. The non-GMP version has also been used in the treatment of two patients with Giant Congenital Melanocytic Nevi (GCMN).The Company is currently completing the final steps for GMP readiness for ESS and thereafter will begin evaluating opportunities to launch a pivotal clinical study with ESS in this areas of GCMN and pediatric severe burns.
About Amarantus BioScience Holdings, Inc.
Amarantus BioScience Holdings (AMBS) is a biotechnology company developing treatments and diagnostics for diseases in the areas of neurology, regenerative medicine and orphan diseases. AMBS' Therapeutics division has development rights to eltoprazine, a Phase 2b-ready small molecule indicated for Parkinson's disease levodopa-induced dyskinesia, adult ADHD and Alzheimer's aggression, and owns the intellectual property rights to a therapeutic protein known as mesencephalic-astrocyte-derived neurotrophic factor (MANF) and is developing MANF-based products as treatments for brain and ophthalmic disorders. More recently, AMBS acquired the rights to the Engineered Skin Substitute program (ESS), a regenerative medicine-based approach for treating severe burns with full thickness autologous skin grown in tissue culture. ESS is entering Phase 2 clinical studies under a CRADA agreement with the US Army. AMBS' Diagnostics division owns the rights to MSPrecise®, a proprietary next-generation DNA sequencing (NGS) assay for the identification of patients with relapsing-remitting multiple sclerosis (RRMS) at first clinical presentation, has an exclusive worldwide license to the Lymphocyte Proliferation test (LymPro Test®) for Alzheimer's disease, which was developed by Prof. Thomas Arendt, Ph.D., from the University of Leipzig, and owns intellectual property for the diagnosis of Parkinson's disease (NuroPro). AMBS also owns the discovery of neurotrophic factors (PhenoGuard™) that led to MANF's discovery.
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